Kv7.2/7.3 Ion Channel Openers

Cell Reports Publishes Discovery Data Supporting the Importance of Ion Channel, Kv7.2/7.3 as a Therapeutic Target in ALS

Retrieved on: 
Tuesday, June 8, 2021

The publication describes a live-cell screening strategy targeting abnormal electrophysiological properties to reveal targets that modulate the intrinsic hyperexcitability of ALS motor neurons.

Key Points: 
  • The publication describes a live-cell screening strategy targeting abnormal electrophysiological properties to reveal targets that modulate the intrinsic hyperexcitability of ALS motor neurons.
  • This unbiased screen using human ALS motor neurons identified Kv7.2/7.3 as a strongly overrepresented drug target.
  • Previous research has identified Kv7.2/7.3 as an ALS drug target and the unbiased nature of this screen further emphasizes the importance of Kv7.2/7.3 in ALS motor neuron dysfunction.
  • We are advancing three antisense and small molecule programs addressing sub-forms of ALS that account for the majority of ALS patients.